ESPN 54th Annual Meeting

ESPN 2022


 
EARLY PRO-TECT Alport XXL: A Worldwide Observational Study to Improve Evidence for Preemptive Start of ACE Inhibitor Therapy in Children With Alport Syndrome
OLIVER GROSS 1 JAN BOECKHAUS 1

1- UNIVERSITY MEDICINE GOETTINGEN, NEPHROLOGY AND RHEUMATOLOGY
 
Introduction:

Children with the type IV collagen disease Alport syndrome (AS) develop renal failure early in life. ACE-inhibition is the first-line off-label therapy and can delay renal failure in a time dependent manner. The EARLY PRO-TECT Alport trial was the first randomized and placebo-controlled trial to evaluate safety and efficacy of ACE-inhibition in children with AS. The trial indicated safety and efficacy of nephroprotective therapy without statistical significance.The aim of this observational study is to analyse renal outcome and adverse effects in the longterm observation of children from the EARLY PRO-TECT Alport trial as well as other children in disease stages 0,I,II from all over the world.

Material and methods:

Supported by the EARLY PRO-TECT Alport XXL team international and the study group of the German Society of Pediatric Nephrology, we developed a predefined study protocol:

Alport Syndrome disease stages are defined as:

0 Microhaematuria without microalbuminuria

I Microalbuminuria (30-300 mg albumin/gCrea)

II Proteinuria >300 mg albumin/gCrea

Primary Endpoint: time to progress of AS to the next disease stage under treatment

Secondary Endpoint: Time to progress of AS to the next disease stage under treatment compared to children without treatment.

Results:

In addition to the 14 German trial sites of EARLY PRO-TECT Alport, we contacted our international cooperation partners with leading pediatric nephrology centers in the US, UK, South Korea, and China, using University based individual cooperation contracts. We cordially invite all pediatric nephrologists to contribute to this international effort. At present, we have data of long-term outcome of more than 150 children in our system. Data collection will end in 2023. 

Conclusions:

PLEASE JOIN OUR INTERNATIONAL EFFORT! Using a Bayesian evidence synthesis approach, different levels of evidence will contribute to enrich the EARLY PRO-TECT Alport data in order to reach the significant levels for efficacy of pre-emptive start of ACE-inhibitor therapy in children with AS. Our international approach also aims to set up a political statement for the worldwide need for early diagnosis of children with AS.